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Aldose Reductase Inhibitor
Epalrestat for PMM2-CDG
Phase 3
Waitlist Available
Led By Eva Morava-Kozicz, MD, PhD
Research Sponsored by Eva Morava-Kozicz
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Age ≥ 2 and < 18 years
Diagnosis of PMM2-CDG, based on molecularly confirmed biallelic PMM2 pathogenic variants (can be historical diagnosis with lab report on file)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 9 months
Awards & highlights
Study Summary
This trialwill test if a drug can help kids with a rare genetic disorder. It's a safe and controlled study.
Who is the study for?
This trial is for children aged 2 to under 18 with PMM2-CDG, a rare genetic disorder. They must be able to follow the study plan and not be pregnant or at risk of pregnancy without proper contraception. Kids can't join if they're allergic to epalrestat, have anemia, kidney problems, low platelets, liver issues, other CDGs or are on certain other drugs.Check my eligibility
What is being tested?
The study tests oral Epalrestat against a placebo in kids with PMM2-CDG. It's designed to see if it's safe and tolerable and whether it improves their condition. Participants will randomly receive either the drug or placebo without knowing which one they get.See study design
What are the potential side effects?
While specific side effects for this trial aren't listed, common ones from similar trials may include allergic reactions to medication ingredients and potential impacts on blood sugar levels due to Epalrestat’s mechanism of action.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 2 and 17 years old.
Select...
I have been diagnosed with PMM2-CDG based on genetic testing.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 9 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~9 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in Antithrombin III (ATIII)
Change in ICARS
Change in sorbitol (mmol/mol creatinine)
Secondary outcome measures
Change in Nijmegen Pediatric CDG Rating Scale (NPCRS) score
Change of Body Max Index (BMI) percentile
Change of factor XI activity percentage
+3 moreTrial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: EpalrestatExperimental Treatment1 Intervention
Epalrestat will be administered orally, 3 times per day (TID) spaced out as evenly as possible over 24 hours in a divided dose starting on Day 1 of the Study.
Group II: PlaceboPlacebo Group1 Intervention
Placebo will be administered orally, 3 times per day (TID) spaced out as evenly as possible over 24 hours in a divided dose starting on Day 1 of the Study.
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Who is running the clinical trial?
Eva Morava-KoziczLead Sponsor
3 Previous Clinical Trials
47 Total Patients Enrolled
Maggie's Pearl, LLCLead Sponsor
Eva Morava-Kozicz, MD, PhDPrincipal InvestigatorMayo Clinic
8 Previous Clinical Trials
1,040 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had liver problems, like high enzyme levels or liver stiffness, in the last 6 months.I am between 2 and 17 years old.I haven't used any experimental drugs or biologics recently.Female participants who could become pregnant need to have a negative pregnancy test.My kidney function is impaired based on my age-specific creatinine levels.Your hemoglobin levels are lower than 10 grams per deciliter.If the doctor thinks any other important lab test results are not normal, you may not be able to join the study.I have or might have a congenital disorder of glycosylation.You are allergic to aldose reductase inhibitors.You are allergic to epalrestat.Your blood platelet count is lower than 125 billion per liter.I have been diagnosed with PMM2-CDG based on genetic testing.
Research Study Groups:
This trial has the following groups:- Group 1: Epalrestat
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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